Following the Data

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Capstone Therapeutics Corp. and its joint development partner, LipimetiX Development, LLC, reported data from its Phase 1b/2a human clinical trials in December. Even at low dose levels, AEM-28 showed statistically significant and substantial reductions in VLDL cholesterol and triglycerides vs. control. The effect in dropping VLDL and TGs was rapid and dramatic: within the first hour post infusion VLDL and TGs plummeted by an approximate average of 70% from base line! Our science advisory board and other physicians and experts in the field have commented that they know of no other drug in the market or being developed that can achieve the same effect.

Experienced drug developers “Follow The Data” and learn from biomarkers what the study drug is really accomplishing pharmacologically. Our SAB suggested “LipimetiX should be going after acute pancreatitis with high triglycerides.” So, we began to aggressively research this new potential indication.

We retained a respected consultancy, Fletcher Spaght (Boston), to conduct a market assessment study for AEM-28 in acute pancreatitis with high triglycerides. We developed a detailed list of interview questions to help determine the market opportunity. Fletcher Spaght then interviewed 15 Key Opinion Leaders (KOLs) including lipidologists, cardiologists and endocrinologists from leading national hospitals. The report concluded that the AP indication represents “a significant unmet clinical need for a therapeutic that could rapidly reduce triglycerides”.

The schedule that follows below discusses the epidemiology and etiology of acute pancreatitis. Main causes are gallstones, alcohol, genetic hypertriglyceridemia and other origins. The KOLs believe all etiologies, except for gallstones, present with high triglycerides. Regardless of cause, elevated triglycerides result in worse outcomes for these patients. According to a highly regarded pancreatologist, the ability to therapeutically reduce TGs within the first 24 hours of onset assists in limiting the damaging inflammatory sequence and pancreatic necrosis. This could result in better patient outcomes and shorter hospital stays. Fletcher Spaght estimates there are approximately 74,000 hospitalizations for all types of acute pancreatitis in the US each year with approximately 45,000 presenting with severe TGs equal to or greater than 1,000 mg/dL. That patient population is an ideal fit for AEM-28.

Whereas acute pancreatitis with high TGs qualifies as an Orphan indication, it is nonetheless a sizable Orphan market. Clinicians treat these patients with hospitalization in acute or ICU care, no food by mouth, a no fat parenteral diet, opiates to address the severe pain and fibrates/fish oil to reduce TGs. The problem is that fibrates and fish oil take weeks if not longer to have an effect in reducing TGs; yet, they are the current standard of care. A drug, such as AEM-28, which rapidly reduces TGs could diminish the severity of AP (especially administered at early onset) and could offer a significant economic savings to the healthcare system from faster discharge. The pancreatologist we consulted suggested treatment with AEM-28 be added to the AP treatment protocol in the emergency room for patients with elevated TGs.

Fletcher Spaght also believes that a market of 110,000 refractory hypertriglyceridemics exists in the US. These patients are at high risk for AP and other TG-related indications and would be candidates for a weekly infusion of a TG-reducing therapeutic such as AEM-28. At a projected 5.7 million doses annually, this chronic market represents another significant market opportunity, albeit requiring clinical outcomes studies.

Given the above, we are excited to prioritize AP with high TGs as our indication of choice for AEM-28 commercialization. As an Orphan indication, the clinical/regulatory pathway is well established and should require shorter, less expensive clinical trials. We continue to believe that the homozygous familial hypercholesterolemia market (HoFH), remains underserved and is also an ideal candidate market for our drug.

We believe we have found a special opportunity in AP, with a clear unmet need, and are quite excited to pursue this indication with our new composition of matter, 21-year commercial life molecule, AEM-28-02.

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Statements in this blog post or otherwise attributable to Capstone regarding our business that are not historical facts are forward looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from predicted results. These risks include the factors discussed in our Form 10-K for the fiscal year ended December 31, 2014, and other documents we file with the U.S. Securities and Exchange Commission.

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